Sustainable approaches for drug repurposing in rare diseases: recommendations from the IRDiRC Task Force

Drug repurposing represents a real opportunity to address unmet needs and improve the lives of rare disease patients. It is often presented as a faster, safer and cheaper path for bringing drugs into new indications. However, several economic, regulatory and scientific barriers can impede the successful repurposing of drugs for rare diseases. The International Rare Diseases Research Consortium (IRDiRC) set up the Task Force on Sustainable Models in Drug Repurposing with the objective of identifying key factors for achieving sustainable repurposing approaches in rare diseases. In order to help inform expert opinion, the Task Force investigated six cases of medicinal products repurposed into new rare indications and four cases of ongoing development programs. A questionnaire addressing the major steps of the repurposing approach was developed by the Task Force and sent to contact points of the organizations. In addition, interviews were conducted with the relevant organization representatives to conduct a deeper dive into the sustainability of the repurposing approach for each of the selected cases. Based on the collective experience of the members of the Task Force and the output from the questionnaires/interviews, we have identified ten key factors that should be considered by those embarking on repurposing projects. These factors include the identification of unmet patient needs and partnership with patients, collection of evidence concerning disease prevalence, patient numbers, drug pharmacology and disease etiology, drug industrial property status, off-label or compounding use, data from past clinical studies and needs for extended non-clinical and clinical studies. The development of a collaborative funding framework and early discussion with regulators and payers are additional factors to implement early in the development of sustainable drug repurposing projects

A Rare Disease Patient Manager

ABSTRACT publicado: 6th International Conference on Practical Applications of Computational Biology & Bioinformatics (PACBB. Salamanca, 28-30 Março 2012The personal health implications behind rare diseases are seldom considered in widespread medical care. The low incidence rate and complex treatment process makes rare disease research an underrated field in the life sciences. However, it is in these particular conditions that the strongest relations between genotypes and phenotypes are identified. The rare disease patient manager, detailed in this manuscript, presents an innovative perspective for a patient-centric portal integrating genetic and medical data. With this strategy, patient’s digital records are transparently integrated and connected to wet-lab genetics research in a seamless working environment. The resulting knowledge base offers multiple data views, geared towards medical staff, with patient treatment and monitoring data; genetics researchers, through a custom locus-specific database; and patients, who for once play an active role in their treatment and rare diseases research

De l'inefficacité du réseau social : des liens sociaux non mobilisés chez les patients atteints de cancer

International audienceDe nombreuses études sur les réseaux sociaux portent sur la question des ressources auxquelles un individu peut avoir accès à travers ses relations sociales. En s'intéressant aux réseaux qui ont « fonctionné », elles tentent alors de spécifier les facteurs ayant permis l'accès aux ressources. Mais peu de travaux se sont penchés sur les réseaux ayant échoué à fournir à l'individu le bien ou le service qu'il recherchait. Cet article se propose de discuter de ces réseaux « inefficaces » à travers le cas de patients atteints de cancer recherchant des informations relatives à leur pathologie. Les résultats indiquent que les causes de l'inefficacité du réseau sont à rechercher dans la non mobilisation des liens de la part des malades rencontrés. Un certain nombre de contraintes pèsent sur ces liens empêchant ainsi leur mobilisation

Health information seeking on the Internet: a double divide? Results from a representative survey in the Paris metropolitan area, France, 2005–2006

<p>Abstract</p> <p>Background</p> <p>The Internet is a major source of information for professionals and the general public, especially in the field of health. However, despite ever-increasing connection rates, a digital divide persists in the industrialised countries. The objective of this study was to assess the determinants involved in: 1) having or not having Internet access; and 2) using or not using the Internet to obtain health information.</p> <p>Methods</p> <p>A cross-sectional survey of a representative random sample was conducted in the Paris metropolitan area, France, in the fall of 2005 (n = 3023).</p> <p>Results</p> <p>Close to 70% of the adult population had Internet access, and 49% of Internet users had previously searched for medical information. Economic and social disparities observed in online health information seeking are reinforced by the economic and social disparities in Internet access, hence a double divide. While individuals who reported having a recent health problem were less likely to have Internet access (odds ratio (OR): 0.72, 95% confidence interval (CI): 0.53–0.98), it is they who, when they have Internet access, are the most likely to search for health information (OR = 1.44, 95% CI = 1.11–1.87).</p> <p>Conclusion</p> <p>In the French context of universal health insurance, access to the Internet varies according to social and socioeconomic status and health status, and its use for health information seeking varies also with health beliefs, but not to health insurance coverage or health-care utilisation. Certain economic and social inequalities seem to impact cumulatively on Internet access and on the use of the Internet for health information seeking. It is not obvious that the Internet is a special information tool for primary prevention in people who are the furthest removed from health concerns. However, the Internet appears to be a useful complement for secondary prevention, especially for better understanding health problems or enhancing therapeutic compliance.</p

Health Research on Immunization after 2016: the need for a plural methodological approach

In French : https://hal.archives-ouvertes.fr/hal-03675482International audienceThe current “generalized digitization” of society is influencing the health environment, healthcare organizations as well as actors. In this context, human and social sciences deconstruct, nuance and sometimes even challenge certain preconceived ideas and/or dominant discourses.In this book, researchers of four nationalities and three different disciplines have agreed to open the “black box” of their work. They display their scientific practices from the perspective of epistemology, ethics and methodology. They present and analyze their values and postulates but, also, what may have influenced the project, the definition of the object and objectives, as well as their approaches. In a contextual way, the first part presents some changes in environments and info-communicational practices related to digital health. The second part opens space to reflect on ethics and deontology. Finally, postulating that scientific fact is not an essence but the result of a process, the last part discusses the methods implemented, which may be different from those initially envisaged.This book is dedicated to the researchers and postgraduate students in the human and social sciences as well as the health practitioners likely to collaborate with them